THOC6-related developmental delay-microcephaly-facial dysmorphism syndrome

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ORPHA:363444OMIM:613680Q87.0
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Overview

THOC6-related developmental delay-microcephaly-facial dysmorphism syndrome is a rare genetic condition caused by changes (mutations) in the THOC6 gene. This gene plays an important role in how cells process and transport genetic instructions (called mRNA), which is essential for normal brain development and growth. The condition is also sometimes referred to as Beaulieu-Boycott-Innes syndrome (BBIS). Children born with this syndrome typically have a smaller-than-average head size (microcephaly), intellectual disability ranging from mild to severe, and distinctive facial features. These facial features may include a flat midface, a short nose, widely spaced eyes, and a thin upper lip. Many affected children also experience delays in reaching developmental milestones such as sitting, walking, and talking. Some individuals may have heart defects, kidney abnormalities, or other organ involvement. There is currently no cure for this condition. Treatment focuses on managing symptoms and supporting development. This includes early intervention programs, speech therapy, occupational therapy, physical therapy, and special education services. Heart or kidney problems, if present, are treated by the appropriate specialists. With supportive care, many individuals can make meaningful developmental progress, though most will need some level of lifelong support.

Also known as:

Key symptoms:

Smaller than average head size (microcephaly)Intellectual disabilityDelayed speech and language developmentDelayed motor milestones like sitting and walkingDistinctive facial features such as a flat midface and short noseWidely spaced eyesThin upper lipHeart defects present at birthKidney abnormalitiesShort stature or poor growthLow muscle tone in infancyFeeding difficulties in early lifeSeizures in some individualsBehavioral challengesVision problems

Clinical phenotype terms (44)— hover any for plain English
Long noseHP:0003189Low hanging columellaHP:0009765Mild microcephalyHP:0040196Dysplastic corpus callosumHP:0006989
Inheritance

Autosomal recessive

Passed on when both parents carry the same gene change; often skips generations

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for THOC6-related developmental delay-microcephaly-facial dysmorphism syndrome.

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No actively recruiting trials found for THOC6-related developmental delay-microcephaly-facial dysmorphism syndrome at this time.

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Search ClinicalTrials.gov ↗Join the THOC6-related developmental delay-microcephaly-facial dysmorphism syndrome community →

No specialists are currently listed for THOC6-related developmental delay-microcephaly-facial dysmorphism syndrome.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to THOC6-related developmental delay-microcephaly-facial dysmorphism syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Caregiver Resources

NORD Caregiver Resources

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Mental Health Support

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Family & Caregiver Grants

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Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Questions for your doctor

Bring these to your next appointment

  • Q1.What is the specific genetic change found in my child's THOC6 gene, and what does it mean for their outlook?,Should my child have heart and kidney screening, and how often should these be repeated?,What early intervention therapies do you recommend, and how soon should we start?,Are there any clinical trials or research studies my child might be eligible for?,What should I watch for in terms of seizures, and what should I do if one occurs?,Should other family members be tested to see if they are carriers?,What resources or support groups are available for families affected by this condition?

Common questions about THOC6-related developmental delay-microcephaly-facial dysmorphism syndrome

What is THOC6-related developmental delay-microcephaly-facial dysmorphism syndrome?

THOC6-related developmental delay-microcephaly-facial dysmorphism syndrome is a rare genetic condition caused by changes (mutations) in the THOC6 gene. This gene plays an important role in how cells process and transport genetic instructions (called mRNA), which is essential for normal brain development and growth. The condition is also sometimes referred to as Beaulieu-Boycott-Innes syndrome (BBIS). Children born with this syndrome typically have a smaller-than-average head size (microcephaly), intellectual disability ranging from mild to severe, and distinctive facial features. These facial

How is THOC6-related developmental delay-microcephaly-facial dysmorphism syndrome inherited?

THOC6-related developmental delay-microcephaly-facial dysmorphism syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does THOC6-related developmental delay-microcephaly-facial dysmorphism syndrome typically begin?

Typical onset of THOC6-related developmental delay-microcephaly-facial dysmorphism syndrome is neonatal. Age of onset can vary across affected individuals.